The Radiopharmaceutical Therapy and Dosimetry Lab at Johns Hopkins Medicine, headed by George Sgouros, Ph.D., has been awarded a $15 million grant, to be dispersed over the next five years, from the National Cancer Institute at the National Institutes of Health. They will use these funds to investigate a type of radiation therapy for hard-to-treat cancers. The award will fund several research projects, all of which will investigate a promising cancer treatment known as alpha-particle emitter radiopharmaceutical therapy, or alpha-emitter RPT. This therapy has proved successful in treating widespread, treatment-resistant cancers through directly delivering radiation to cancer cells. Administered into the bloodstream, alpha-emitter RPT treatment delivers highly potent radiation directly to cancer cells. Radioactive atoms that emit alpha-particles (helium nuclei) are attached to special molecules that target, or latch on to, dispersed cancer cells in the body. Alpha-particles delivered to cancer cells cause massive DNA damage that kills the cancer. ...
Recently, the “Pyrotinib Combined with Docetaxel as First-Line Treatment for HER2-Positive Metastatic Breast Cancer: An Open, Multicenter Phase II Clinical Study (PANDORA)” led by Professor Wang Xiaojia from the Affiliated Cancer Hospital of the University of Chinese Academy of Sciences (Zhejiang Cancer Hospital) was officially published in the internationally renowned journal “Nature Communications,” a sub-journal of ‘Nature’ (Impact Factor: 16.6). The research results indicate that the combination therapy of pyrotinib and docetaxel demonstrates excellent anti-tumor activity and brings about progression-free survival (PFS) benefits. Research Background: Dual-targeted therapy with trastuzumab combined with taxanes has established the first-line standard treatment for HER2-positive advanced breast cancer [Progression-Free Survival (PFS): 18.7m vs. 12.4m, P<0.001; 8-year Overall Survival (OS) rate: 37% vs. 23%, P<0.001]. However, the inclusion of trastuzumab in the Cleopatra study in a population with prior trastuzumab (referred to as “H”) treatment was only 11%, and it may no longer fully guide current ...
SHANGHAI and CLINTON (NJ), December 19, 2023 – HuidaGene Therapeutics (辉大基因“HuidaGene”) today announced the U.S. FDA granted Rare Pediatric Drug Designation (RPDD) to HG302, a novel CRISPR-Cas12 DNA-editing therapy, to treat Duchenne muscular dystrophy (DMD), affecting 1 in ~5,000 newborn boys. Unlike the in-vivo use of Cas9 system with limited genome-editing efficiency, high occurrence of off-targeting modifications, viral delivery packaging limitations, and complicated CRISPR-Cas9 patent dispute, HuidaGene identified novel Cas12i proteins using AI and deep machine-learning of DNA sequencing through the proprietary HG-PRECISE® platform to develop high-fidelity Cas12i variant (hfCas12Max®). This hfCas12Max® is smaller in size than Cas9, thereby packaging it using one viral vector with the lowest off-targeting activity and highest editing efficiency in mammalian cells when compared to SpCas9 and LbCas12a. Furthermore, Cas12i was granted a patent by USPTO with a cleaner commercial landscape. “This is Company’s third program receiving RPDD, suggesting the recognition of the importance of ...
It’s time to build the infrastructure needed to scale up inhaled gene therapies targeting cystic fibrosis, and to bolster investment that supports several key program components. By JOAN LAU Thanks to simultaneous achievements in gene therapy and medicine delivery platforms, new solutions to intractable problems are bringing us to a real watershed moment in the treatment of respiratory disease. This synchronicity of science and tech advancements is no accident; along with colleagues in my own and other organizations, I have been working for the past several years on the medical and technological leaps that together signal hope for respiratory-disease patients with unmet need. This is especially true for those with cystic fibrosis (CF), for whom the standard of care is in urgent need of update. And now that we have the science and the technology in place, we must push to scale-up efforts; patients are waiting. Right now, CF patients ...
Despite bluebird bio winning a recent FDA nod for its much-anticipated sickle cell disease gene therapy Lyfgenia, the regulatory triumph left something to be desired for the company because the agency didn’t grant a lucrative priority review voucher (PRV). That came as a surprise to bluebird, which had previously inked a deal to sell a potential PRV to Novartis for $103 million. Now, bluebird is hurrying to raise millions of dollars—through two separate avenues—to fund the launch of its new sickle cell disease gene therapy and others. In one fundraising approach, bluebird said it reached an agreement to hand over future gene therapy revenues to Alterna Capital Solutions in exchange for up-front funding. The deal covers a maximum credit of $100 million for bluebird, the company said in an SEC filing (PDF). Additionally, bluebird is working with Goldman Sachs and J.P. Morgan Securities to sell $150 million in new shares. ...
Despite achieving its Phase 3 study’s main goal, the results for Point Biopharma’s therapy are short of what Novartis radiopharmaceutical Pluvicto achieved in a similarly designed clinical trial. Nevertheless, the data readout should clear the way for Eli Lilly to complete its $1.4 billion acquisition of Point. By FRANK VINLUAN A Point Biopharma Global cancer therapy key to a $1.4 billion Eli Lilly M&A deal has met the main goal of its pivotal clinical trial in prostate cancer. But the results raise questions about how well the drug will match up against a radiopharmaceutical marketed by Novartis. The Point drug, PNT2002, is also a radiopharmaceutical. It offers targeted delivery of a radioactive particle by linking it to a ligand that targets PSMA, a protein abundant on the surface of prostate cancer cells. The open-label Phase 3 study evaluated the Point radiopharmaceutical in patients with cases of prostate cancer that has ...
On December 15, 2023, BIO-THERA Bio-Pharmaceutical Co., Ltd. announced that it had received a Notice of Approval of Drug Clinical Trial from the State Drug Administration, which approved the application for a Phase II/III clinical trial of the investigational drug BAT1308 injection in combination with platinum-containing chemotherapy for the first-line treatment of advanced or recurrent mismatch-modification-repaired protein-deficient (dMMR) endometrial cancer. BAT1308 is a humanized anti-PD-1 monoclonal antibody independently developed by BIO-THERA, which belongs to IgG4κ subtype and is expressed by Chinese hamster ovary cells.PD-1 is mainly expressed by activated T-lymphocytes, and is an inhibitory immune checkpoint.PD-L1 and PD-L2 are the two ligands of PD-1, and when PD-L1 or PD-L2 bind to PD-1, it can inhibit immune activation of T-cells through the downstream signaling pathway to inhibit immune activation of T cells. It has been found that a variety of tumor cells can express PD-L1 and PD-L2 and bind to PD-1 ...
Despite the high list price of Lyfgenia, bluebird has signed a large reimbursement deal for the sickle cell disease (SCD) gene therapy less than a week after its FDA approval. The outcomes-based agreement bluebird reached for Lyfgenia is with an organization that covers about 100 million people in the U.S., the gene therapy specialist said in a securities filing (PDF) Thursday. That’s nearly a third of the country’s entire population, which is estimated at 335 million. For Lyfgenia, bluebird is offering an outcomes-based framework that tracks treatment results for three years and offers rebates if a patient is hospitalized because of a painful episode of vaso-occlusion, which is a common complication of SCD. The deal marks Lyfgenia’s first coverage agreement. A bluebird spokesperson declined to disclose more details of the contract for competitive reasons. Meanwhile, the speed of this deal underscores that payers recognize the value of the therapy and ...
Pharmaceutical Executive Editorial Staff MAPS Public Benefit Corporation filed a new drug application for MDMA (midomafetamine capsules) for use with psychological intervention for the treatment of post-traumatic stress disorder. MAPS Public Benefit Corporation has submitted a new drug application (NDA) to the FDA in what would represent the first approved psychedelic treatment for post-traumatic stress disorder (PTSD). MAPS is seeking approval of MDMA (midomafetamine capsules) for use with psychological intervention comprised of psychotherapy and additional supportive services provided by a qualified healthcare provider for PTSD.1 MAPS was previously granted Breakthrough Therapy Designation by the FDA for MDMA and is seeking a priority review for the NDA submission. “The filing of our NDA is the culmination of more than 30 years of clinical research, advocacy, collaboration and dedication to bring a potential new option to adults living with PTSD, a patient group that has experienced little innovation in decades,” Amy Emerson, ...
Last month, Bristol Myers Squibb made the surprise announcement that the FDA will convene an advisory committee meeting to discuss the company’s bid to move the CAR-T therapy Abecma into earlier lines of treatment. Now, we know at least one reason why the FDA wants more opinions on the application. Abecma showed no advantage in extending patients’ lives compared with standard combination regimens when used in multiple myeloma patients after two to four prior lines of therapy, according to an updated interim analysis of the phase 3 KarMMa-3 trial presented at the 65th American Society of Hematology annual meeting. The statistical analysis revealed a negligible 1% increased risk of death for Abecma. But median overall survival looks better for Abecma, as patients lived a median of 41.4 months versus 37.9 months for control. The lack of an overall survival showing came in stark contrast to the 51% reduction in the ...
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